Core Insights - Ultragenyx Pharmaceutical reported a second-quarter 2025 loss of $1.17 per share, which is an improvement from a loss of $1.52 per share in the same quarter last year and better than the Zacks Consensus Estimate of a loss of $1.27 [1][5] - Total revenues for the quarter reached $166.5 million, reflecting a 13% year-over-year increase, driven primarily by higher product sales, and surpassing the Zacks Consensus Estimate of $162 million [1][5] Revenue Breakdown - Crysvita generated total revenues of $120.4 million, up 6% year over year, with contributions of $79 million from North America, $35 million from Latin America and Turkey, and $7 million from Europe [3] - Mepsevii product revenues increased by 35% year over year to $8.3 million, while Dojolvi revenues rose 20% to $23.2 million due to new patient demand [4] - Evkeeza recorded sales of $14.6 million in the first quarter, showing significant growth as the drug continues to be launched in territories outside the United States [4] Financial Guidance - The company reaffirmed its 2025 financial guidance, expecting total revenues between $640 million and $670 million, which represents a growth of approximately 14-20% compared to 2024 [9] - Crysvita revenues are anticipated to be in the range of $460-$480 million, reflecting a year-over-year increase of 12-17%, while Dojolvi revenues are expected to be between $90 million and $100 million, up 2-14% year over year [9] Operating Expenses - Operating expenses for the quarter were $274.4 million, a 4% increase year over year, attributed to higher investments in late-stage pipeline programs and marketing costs for approved drugs [7] - Research and development expenses were $164.7 million (up 2%), selling, general and administrative expenses were $86.6 million (up 7%), and cost of sales was $23 million (up 8%) [7] Pipeline Updates - The FDA issued a complete response letter for Ultragenyx's biologics license application for UX111, requesting additional information related to manufacturing processes, which the company plans to address promptly [11][12] - The company is also developing GTX-102 for Angelman syndrome, which received Breakthrough Therapy designation, with data expected in the second half of 2026 [14] - Ultragenyx plans to submit a BLA for DTX401, a gene therapy for glycogen storage disease type Ia, in the fourth quarter of 2025 [15]

NEW YORK, Aug. 6, 2025 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Mereo BioPharma Group plc ("Mereo" or the "Company") (NASDAQ: MREO).  Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980.  The investigation concerns whether Mereo and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about joining the class action]On July 9, 2025, Me ...

Core Viewpoint - HC Wainwright has initiated coverage on Ultragenyx Pharmaceuticals, Inc. with a Buy rating and a price target of $80, highlighting it as an attractive investment opportunity despite a 35% year-to-date decline in its stock price compared to a 2.7% decline in the XBI index [1][2]. Group 1: Company Performance and Trials - Ultragenyx's stock has faced pressure primarily due to investor disappointment regarding the interim results from the pivotal Orbit trial of UX143 (setrusumab) for osteogenesis imperfecta [2]. - The Phase 3 portion of the Orbit study is progressing towards a final analysis by the end of the year, with the second interim analysis not meeting the minimal p-value threshold to conclude the study early [3]. - The analyst believes the Orbit trial has not failed, and positive final results are anticipated, which should support a U.S. regulatory filing for setrusumab next year [4]. Group 2: Commercial Portfolio and Future Prospects - Ultragenyx's current commercial portfolio is estimated to support a fair value of approximately $35-$40 per share, excluding the potential contributions from its pipeline [4]. - The flagship Crysvita franchise is projected to achieve peak annual sales exceeding $3 billion [5]. - The company is expected to resubmit its Biologics License Application (BLA) for UX111 (ABO-102) later this year, with potential approval anticipated by mid-2026, addressing a high unmet need in the Sanfilippo syndrome market [6][7].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Mereo BioPharma Group plc and its officers or directors [1] Group 1: Company Developments - On July 9, 2025, Mereo and Ultragenyx Pharmaceutical Inc. announced progress in the Phase 3 Orbit study for UX143 (setrusumab) in treating osteogenesis imperfecta, with a final analysis expected by the end of the year [3] - Following this announcement, Mereo's ADR price dropped by $1.25, or 42.52%, closing at $1.69 per share on July 10, 2025 [3] Group 2: Legal Context - Pomerantz LLP is recognized for its expertise in corporate, securities, and antitrust class litigation, having a long history of fighting for victims of securities fraud and corporate misconduct [4] - The firm has successfully recovered numerous multimillion-dollar damages for class members over its 80-year history [4]

The Pomerantz Firm, with offices in New York, Chicago, Los Angeles, London, and Paris is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean of the class action bar, the Pomerantz Firm pioneered the field of securities class actions. Today, more than 80 years later, the Pomerantz Firm continues in the tradition he established, fighting for the rights of the victims of securities fraud, bre ...

Group 1 - Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. regarding potential securities fraud or unlawful business practices by the company and its officers [1] - On July 9, 2025, Ultragenyx and Mereo BioPharma Group announced the progress of the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta, with a final analysis expected by the end of the year [3] - Following the announcement, Ultragenyx's stock price dropped by $10.41, or 25.11%, closing at $31.04 per share on July 10, 2025 [3] Group 2 - The Pomerantz Firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having pioneered the field of securities class actions over 80 years ago [4] - The firm has a history of recovering multimillion-dollar damages awards for victims of securities fraud and corporate misconduct [4]

Core Viewpoint - Ultragenyx Pharmaceuticals faces a regulatory setback as the FDA issued a Complete Response Letter (CRL) for its biologics license application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA) [1][6]. Regulatory Update - The CRL requested additional information and enhancements related to chemistry, manufacturing, and controls, stemming from recent facility inspections. The issues are facility- and process-related, not linked to product quality, and can be addressed quickly [2][6]. - Ultragenyx plans to collaborate closely with the FDA to resolve the observations and expects to resubmit the BLA, which will initiate a review period of up to six months [2]. Clinical Data and Impact - The FDA acknowledged the robustness of the neurodevelopmental outcome data and supportive biomarker findings. However, updated clinical data from current patients is required for the resubmission [3]. - The regulatory delay has pushed the potential approval of UX111 for MPS IIIA to 2026. Year-to-date, shares of Ultragenyx have decreased by 30.5%, contrasting with a 0.9% decline in the industry [3]. Disease Context - MPS IIIA is a rare, fatal lysosomal storage disease affecting the brain, with no approved treatments available. Approximately 3,000 to 5,000 patients are affected, with a median life expectancy of 15 years [9]. Pipeline Overview - Ultragenyx has several other gene therapy candidates in its pipeline, including UX143 for osteogenesis imperfecta and UX701 for Wilson disease, with ongoing studies showing promising results [11][12]. - The investigational gene therapy UX111 has received multiple designations in the U.S. and EU, including Regenerative Medicine Advanced Therapy and Orphan Drug designations [10].

Core Insights - Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma (MREO) have provided an update on the mid-to-late-stage Orbit study for UX143 (setrusumab) aimed at treating osteogenesis imperfecta (OI) in pediatric and young adult patients, with final analysis expected by the end of 2025 [1][5] - The shares of both companies experienced a significant decline in premarket trading following the announcement of the delay in new data from the Orbit study [2][5] - UX143 is a fully human monoclonal antibody that inhibits sclerostin, and interim safety data has shown an acceptable safety profile, allowing the study to proceed as planned [2][5] Study Details - The Orbit study is assessing the impact of UX143 on fracture rates in patients aged 5 to 25 with OI, with a pivotal phase III portion enrolling 159 additional patients [7] - The primary efficacy endpoint of the Orbit study is to reduce the annualized clinical fracture rate, with participants moving into an open-label extension period after the primary analysis [7] - The Cosmic study targets a younger population aged two to under seven years, comparing UX143 to intravenous bisphosphonates for fracture reduction, with 69 patients enrolled [8] Partnership and Market Context - Ultragenyx Pharmaceuticals and Mereo BioPharma entered a licensing agreement in 2020 for the joint development of UX143, with RARE holding exclusive rights in several regions [9] - The partnership was expanded in 2024 through a manufacturing and supply agreement, with RARE responsible for supplying setrusumab to MREO [9] - OI affects approximately 60,000 individuals in commercially accessible geographies, and there are currently no approved treatments available [11]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. and Mereo BioPharma Group plc stocks are experiencing significant declines following the update on the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta (OI) [1][2]. Group 1: Study Progress and Analysis - The Phase 3 portion of the Orbit study is progressing towards a final analysis expected by the end of the year, in line with the original plan [2]. - The second interim analysis did not meet the minimal p-value threshold of p<0.01, which would have allowed the study to conclude early [3]. - The Data Monitoring Committee confirmed that UX143 has an acceptable safety profile, recommending the continuation of the study [3][4]. Group 2: Future Expectations - The final analyses for the Orbit and Cosmic studies will occur after patients have been on therapy for at least 18 months, with thresholds set at p<0.04 for Orbit and p<0.05 for Cosmic [5]. - Analysts express optimism regarding the final analysis, anticipating a positive outcome due to longer follow-up duration and a lower statistical significance threshold [6]. Group 3: Market Reaction - Following the update, RARE stock has dropped by 26.2% to $30.60, while MREO stock has decreased by 34.7% to $1.919 [7].

Core Insights - Ultragenyx Pharmaceuticals (RARE) has received FDA Breakthrough Therapy designation for its investigational therapy GTX-102 (apazunersen) aimed at treating Angelman syndrome (AS) [1][5]. FDA Breakthrough Therapy Designation - The Breakthrough Therapy designation accelerates the development and review of drugs for serious conditions, granted when early clinical evidence indicates significant improvement over existing treatments [2]. - Drugs with this designation benefit from enhanced guidance and support from senior FDA officials [2]. Clinical Study Results - GTX-102 demonstrated rapid and lasting improvements in AS patients during a phase I/II study lasting up to three years, involving 74 patients aged 4 to 17 with complete maternal UBE3A gene deletion [5][6]. - Participants showed consistent developmental progress and ongoing improvements across multiple symptom areas during treatment [6]. Current and Future Studies - Ultragenyx is currently enrolling patients for a phase III Aspire study and plans to initiate a phase II/III Aurora study to evaluate GTX-102 for a broader range of AS genotypes [8]. - The Aurora study is expected to start in the second half of 2025 [8]. Market Context - AS is a rare neurogenetic disorder affecting approximately 60,000 individuals in accessible markets, leading to severe developmental challenges with no approved therapies currently available [7]. Other Clinical Programs - Ultragenyx is developing other gene therapy candidates, including UX143 for osteogenesis imperfecta, which received Breakthrough Therapy designation in October 2024 [9]. - The company is also evaluating UX701 for Wilson disease and has achieved significant results in the phase III GlucoGene study for glycogen storage disease type Ia [10]. - A regulatory application for UX111, an AAV gene therapy for Sanfilippo syndrome type A, is under Priority Review, with a decision expected on August 18, 2025 [11].